rAAV9-cytoplasmic JDP

rAAV9-cytoplasmic JDP is an experimental gene therapy designed to treat tauopathies, including Alzheimer's disease, by delivering a transgene encoding a cytoplasmic J-domain protein (JDP). JDPs are molecular chaperones that facilitate the restoration of healthy protein conformations and the clearance of damaged proteins. In preclinical studies using the PS19 mouse model of tauopathy, hippocampal administration of this recombinant adeno-associated virus serotype 9 (AAV9) vector led to a significant reduction in pathological tau seeding activity. The therapy appears to work by stabilizing the native conformation of the microtubule-associated protein tau (tau), thereby preventing its misfolding and subsequent aggregation without altering overall tau protein levels. This research is being conducted at the University of Texas Southwestern Medical Center.

Other names

rAAV9-JDPrAAV-9-JDPrAAV 9-JDP